Session: 908. Outcomes Research: Myeloid Malignancies: Poster I
Hematology Disease Topics & Pathways:
Research, Acute Myeloid Malignancies, AML, Clinical Practice (Health Services and Quality), Clinical Research, Diseases, Real-world evidence, Myeloid Malignancies
Methods: This was a retrospective, observational study using data extracted from the medical records of pts with AML in remission after induction/consolidation therapy and treated with Oral-AZA maintenance therapy in England and Germany. Oral-AZA must have been initiated between June 17, 2021, and June 20, 2023; data were extracted April–May 2024. Pts who had participated in a clinical trial assessing investigational products or who had received HSCT before Oral-AZA were ineligible. Pt characteristics, treatment patterns, relapse, survival, and occurrence of adverse events (AEs) during Oral-AZA treatment were analyzed descriptively. Kaplan-Meier methods were used to estimate time-to-event outcomes (ie, OS and real-world RFS [rwRFS]).
Results: Data were extracted for 208 pts (England, n=108; Germany, n=100). Most pts were White (82%) and male (61%), with a median age of 67.8 years (range, 31.0–83.9) at Oral-AZA initiation; 11% of pts were <55 years. The Eastern Cooperative Oncology Group performance status score was assessed for most pts (n=163; 78%) and was primarily 0 (37%) or 1 (43%). Regarding disease classification, 87% of pts were diagnosed with de novo AML. Genetic risk status assessed using the 2022 European LeukemiaNet (ELN) classification was available for 138 pts (66%) and was favorable for 65 pts (47%), intermediate for 51 pts (37%), and adverse for 22 pts (16%). The most common gene mutations at diagnosis were NPM1 (16%), RUNX1 (9%), ASXL1 (8%), and TP53 (8%). Overall, 78% of pts were treated with intensive chemotherapy induction regimens, with cytarabine + anthracycline “7+3” being the most common (55%), followed by CPX-351 (7%). Venetoclax-based regimens were given to 10% of pts. One-third (n=68; 33%) of pts received consolidation regimens, with a median of 2 cycles; 35% (n=24/68) received ≥3 cycles.
After a median follow-up of 12.8 months (mo), treatment with Oral-AZA was still ongoing for 137/208 pts (66%). The median duration of Oral-AZA treatment was 10.8 mo (range, 0.2–34.1); 6% of pts received Oral-AZA for 1–3 mo, 10% for 4–6 mo; 44% for 7–11 mo, and 39% for ≥1 year. Most pts (76%) began Oral-AZA at 300 mg for 14 days/cycle, with 69% remaining on this schedule at discontinuation/last visit. Antiemetic medication was provided to 112 pts (54%), in 96% (n=108/112) as prophylaxis. Approximately half (n=122; 59%) of pts had ≥1 AE during Oral-AZA treatment; the most common were nausea (25%) and fatigue (24%).
At 12 mo, estimated OS and rwRFS rates from Oral-AZA initiation were 88.2% (standard error [SE], 2.3) and 85.5% (SE, 2.5), respectively. Median OS and rwRFS were not reached.
Conclusions: Real-world survival outcomes of pts with AML in remission treated with Oral-AZA corroborate findings from the pivotal QUAZAR AML-001 clinical trial. Safety outcomes were consistent with the known safety profile of Oral-AZA. These findings support broad use of Oral‑AZA maintenance therapy in current clinical practice for pts with AML who do not proceed to HSCT at remission.
Disclosures: Krishnamurthy: Jazz Pharmaceuticals: Other: Conference support; AbbVie: Other: Conference support, Speakers Bureau; Gilead: Other: Conference support; Stemline-Menarini: Other: Conference support, Speakers Bureau; Pfizer: Speakers Bureau; Johnson & Johnson: Speakers Bureau; Astellas: Speakers Bureau. Houghton: RTI Health Solutions: Current Employment. Lorenz: Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company; Syndax Pharmaceuticals Inc.: Current equity holder in publicly-traded company; Kura Oncology, Inc.: Current equity holder in publicly-traded company. Williams: Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company. Currie: Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company. Vigil: Bristol Myers Squibb: Current Employment. Ziba: RTI Health Solutions: Current Employment. Bello: RTI Health Solutions: Current Employment. Sieluk: Bristol Myers Squibb: Current Employment, Divested equity in a private or publicly-traded company in the past 24 months. Strocchia: Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company. Li: Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company; Merck & Co.: Current equity holder in publicly-traded company, Ended employment in the past 24 months. Kapp-Schwoerer: Klinikum der Stadt Ludwigshafen: Current Employment; Universitatsklinikum Ulm: Ended employment in the past 24 months; AbbVie: Honoraria; Bristol Myers Squibb: Honoraria; Pfizer: Honoraria; Jazz Pharmaceuticals: Honoraria.
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